Time for Change – Evolution of Real-world Evidence Outcome Measures in Multiple Sclerosis Exemplified by Fingolimod

Abstract

Real-world evidence provides important information concerning the long-term effectiveness and safety of disease-modifying treatments (DMTs) for multiple sclerosis (MS) in clinical practice in a large number of patients. These data enhance and extend the results from randomised clinical trials and include information that cannot easily be obtained in trials such as treatment efficacy in non-trial populations (e.g. those with co-morbidities, older patients and children) and long-term safety analyses. Such data are compatible with specialist clinical practice and, when accumulated in multicentre databases using an agreed minimum dataset, these data can provide invaluable information on long-term disease progression and treatment. Real-world evidence in terms of fingolimod is reviewed and clearly demonstrates its long-term effectiveness and safety. Currently, imaging data are not systematically collected but the development of improved standard magnetic resonance imaging (MRI) protocols and automated procedures now makes this a realistic option in the near future. Automated (or largely automated) MRI analytics are likely to greatly enhance and further strengthen real-world evidence for outcomes of different treatment algorithms incorporating more sophisticated MRI measures.

Fingolimod's Impact on MRI Brain Volume Measures in Multiple Sclerosis: Results from MS‐MRIUS

“…Because the RCTs are designed to include narrowly defined, optimal conditions in specific populations, real‐world studies are necessary to investigate safety and efficacy outcomes of DMTs in a more heterogeneous group's patients, more representative of the overall MS population followed in clinical practice . While the routine assessment of the lesion outcomes is part of standard magnetic resonance imaging (MRI) monitoring in MS, the examination of brain volume changes is limited due to the lack of standardized image acquisition technology and image analysis .…”

Assessing ‘No Evidence of Disease Activity’ Status in Patients with Relapsing-Remitting Multiple Sclerosis Receiving Fingolimod in Routine Clinical Practice: A Retrospective Analysis of the Multiple Sclerosis Clinical and Magnetic Resonance Imaging Outcomes in the USA (MS-MRIUS) Study

“…Furthermore, the frequency of MRIs conducted in RCTs is higher than in clinical practice. Robust real-world studies can, therefore, complement data from RCTs by investigating outcomes in a population of patients who are receiving treatment and being monitored according to routine clinical practice [ 9 , 12 , 13 ].…”

A Propensity Score Matching Study on the Effect of OnabotulinumtoxinA Alone versus Short-Term Psychodynamic Psychotherapy Plus Drug-of-Choice as Preventive Therapy in Chronic Migraine: Effects and Predictive Factors

“…patients with an absolute standard bias > 0.20. PSM was implemented by a logistic regression approach [24][25][26]. Baseline variables with a signi cant difference between groups were chosen as parameters on which to perform the matching rather than using an aprioristic approach.…”